At the annual congress of the American College of Rheumatology, ACR Convergence 2025, specialists presented significant advances in the therapy of Familial Mediterranean Fever (FMF). The data presented confirmed the high efficacy and safety of an innovative interleukin-1 blocker in treating this rare hereditary autoinflammatory disease.
R-Pharm has submitted documents for the registration of goflikicept in Russia for the FMF indication, with the approval expected in 2026. The group of companies plans to conduct an open-label study of the drug for FMF in adolescents aged 12 to 17 years.
FMF is a genetic autoinflammatory disorder causing recurrent fever and pain in the abdomen, chest, and joints, which can lead to kidney-damaging amyloidosis and other organ damage if untreated. Interleukin-1 plays a key role in the disease pathogenesis, making the blockade of this pathway one of the most promising directions in modern therapy.
The researchers presented the results of an international, multicenter, double-blind study, which aimed to evaluate the efficacy and safety of R-Pharm’s drug goflikicept in patients with FMF who are uncontrolled by standard treatments. Participants were randomly assigned to groups receiving either the drug or a placebo. According to the study results, goflikicept significantly improved the condition of patients with FMF compared to placebo, was well-tolerated, and had a safety profile comparable to other IL-1 inhibitors.
The research program for goflikicept in FMF is underway in Russia, Armenia, and Turkey. The double-blind trial has now been completed, and the next phase—a long-term extension study where patients who completed the double-blind trial receive the drug in an open-label manner—has been initiated across all centers.
“R-Pharm is at the forefront of scientific innovation — we work with rare and complex diseases, striving to provide patients with proper treatment. This is confirmed by the data from the international clinical trial of goflikicept therapy in patients with FMF who are unresponsive to standard treatment, presented at ACR Convergence this year by one of the world’s leading experts in the therapy of autoinflammatory diseases — Professor Serdal Ugurlu from Istanbul Medical University. Exchanging data with the international scientific and medical community makes it possible to identify needs and set priorities in drug research and development,” said Mikhail Samsonov, Medical Director of R-Pharm.
Earlier, the results of the goflikicept study in FMF were presented in Japan at the annual international congress of the Asia Pacific League of Associations for Rheumatology (APLAR 2025), highlighting the high regard and interest of the international scientific community in new approaches to the therapy of autoinflammatory diseases.
